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NCT02117713 · ClinicalTrials.gov registry record · Phase 2

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

A Phase 2 study of Alagille Syndrome, sponsored by Mirum Pharmaceuticals.

Completed
Registry status
Phase 2
Development phase
34
Enrollment target
11
Study locations

NCT02117713 is a Phase 2 study of Alagille Syndrome that has completed, run by Mirum Pharmaceuticals. The registered enrollment target is 34 participants, below the 57-participant average among 3 other Alagille Syndrome trials with a reported enrollment target (40% lower). The trial reports 11 study locations across 9 states.

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The verdict

NCT02117713, a Phase 2 study of Alagille Syndrome, has completed, sponsored by Mirum Pharmaceuticals.

COMPLETED
Registry status
Phase 2
Development phase
34 participants
Enrollment target
11
Study locations

Study Summary

This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.

Conditions Studied

Interventions

  • DRUG LUM001 (Maralixibat)

Study Locations (11)

California

  • Children's Hospital Los Angeles - Los Angeles
  • University of California at San Francisco Children's Hospital - San Francisco

Pennsylvania

  • The Children's Hospital of Philadelphia - Philadelphia
  • Children's Hospital of Pittsburgh of UPMC - Pittsburgh

Colorado

  • Children's Hospital Colorado - Aurora

Indiana

  • Riley Hospital for Children - Indianapolis

Ohio

  • Cincinnati Children's Hospital Medical Center - Cincinnati

Texas

  • Baylor College of Medicine/Texas Children's Hospital - Houston

Utah

  • University of Utah - Salt Lake City

Washington

  • Seattle Children's Hospital - Seattle

Trial Details

FieldValue
Enrollment Target 34 participants
Start Date 2015-03-16
Est. Completion 2020-06-01
Phase Phase 2

Sponsor

Mirum Pharmaceuticals

28 total trials

What the Registry Record Tells You About NCT02117713

The ClinicalTrials.gov registry entry for NCT02117713 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 34 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 57-participant average among 3 other Alagille Syndrome trials with a reported enrollment target (40% lower). The listed sponsor is Mirum Pharmaceuticals, which has 28 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Alagille Syndrome appearing as the primary indexed condition, and to 1 intervention - of which LUM001 (Maralixibat) is the first listed.

NCT02117713 reports 11 study locations spanning 9 distinct geographic areas - top geographies include California, Pennsylvania, Colorado.

Frequently Asked Questions

What is clinical trial NCT02117713 about?

NCT02117713 is a clinical study titled "An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome". This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of ...

What is the current status of trial NCT02117713?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 34 participants. The study started on 2015-03-16. Estimated completion is 2020-06-01.

What conditions does trial NCT02117713 study?

This clinical trial studies the following conditions: Alagille Syndrome.

What interventions are being tested in trial NCT02117713?

The interventions under investigation include: LUM001 (Maralixibat) (DRUG).

Who is sponsoring clinical trial NCT02117713?

This trial is sponsored by Mirum Pharmaceuticals, which has 28 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT02117713 being conducted?

This trial has 11 study locations across California, Colorado, Indiana, Ohio, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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